FOR THE PTC124-GD-007-DMD STUDY GROUP

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INTRODUCTION Dystrophinopathy is a rare, severe muscle disorder, and nonsense mutations are found in 13% of cases. Ataluren was developed to enable ribosomal readthrough of premature stop codons in nonsense mutation (nm) genetic disorders. METHODS Randomized, double-blind, placebo-controlled study; males ≥ 5 years with nm-dystrophinopathy received study(More)
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